Sio Gene Therapies
| Company type | Subsidiary |
|---|---|
| Industry | Pharmaceutical |
| Predecessor | Axovant Gene Therapies |
| Founded | October 2014 |
| Founder | Vivek Ramaswamy |
| Defunct | April 5, 2023 |
| Fate | Liquidation and dissolution |
| Headquarters | New York City , United States |
Area served | Global |
Key people | David Hung (CEO 2017–2018), Pavan Cheruvu (CEO from 2018) |
| Products | Gene therapies targeting neurological conditions (including AXO-LENTI-PD, AXO-AAV-GM1, AXO-AAV-GM2) |
| Parent | Roivant Sciences |
Sio Gene Therapies, Inc.—previously known as Axovant Gene Therapies—was a clinical-stage pharmaceutical firm dedicated to creating gene therapies targeting neurological conditions. The company was headquartered in New York City and legally registered in Basel, Switzerland. It was established in October 2014 by Vivek Ramaswamy, a former hedge fund analyst and 2024 Republican presidential hopeful. Sio Gene Therapies functioned as a fully owned subsidiary of Roivant Sciences, which itself was founded in May 2014.
History
In December 2014, Axovant secured the rights to the drug intepirdine from GlaxoSmithKline for $5 million—a modest sum reflecting the drug’s prior failure in four clinical trials. Intepirdine was designed to serve as an adjunct treatment for Alzheimer's disease and dementia with Lewy bodies, representing Axovant's sole therapeutic candidate at that time.
Without advancing any clinical development, Axovant proceeded with its initial public offering in June 2015, generating $315 million—the largest biotech IPO recorded up to that point—buoyed by encouraging phase IIB trial results. Notably, the lock-up period for hedge fund investors was unusually brief at 90 days.
July 2017 brought disappointing news when a phase III trial revealed intepirdine lacked efficacy against Alzheimer's disease, triggering a 75% plunge in Axovant’s stock price within a single trading day. The company faced allegations suggesting it operated as a pump-and-dump scheme. Subsequent trials targeting dementia with Lewy bodies also failed, prompting the cessation of intepirdine’s development by 2018.
Axovant further pursued development of nelotanserin, a compound acquired from Roivant and originally developed by Arena Pharmaceuticals, aimed at treating Lewy body dementia. However, after missing its primary endpoint in a late 2018 phase 2 trial for REM sleep behavior disorder, nelotanserin’s development was discontinued.
In 2016, Axovant collaborated with NFL commentator Solomon Wilcots to promote awareness of Alzheimer's clinical studies and sponsored performances of "Forget Me Not," an initiative focusing on increasing participation from African American communities historically underrepresented in medical research.
David Hung assumed the role of CEO in 2017 but resigned the following year; he was succeeded by Pavan Cheruvu.
By December 2018, Axovant broadened its focus to include gene therapies by licensing treatments for GM1 gangliosidosis as well as Tay–Sachs and Sandhoff diseases. In June 2019, the company partnered with contract manufacturer Yposkesi to enhance its gene therapy production capabilities. Toward the end of 2019, Axovant prepared to disclose data from three clinical-stage programs: AXO-LENTI-PD and the gene therapies AXO-AAV-GM1 and AXO-AAV-GM2.
In November 2020, Axovant underwent rebranding and adopted the name Sio Gene Therapies. On April 5, 2023, shareholders approved the company's liquidation and formal dissolution.